Recruiting

VamoroloneRegistry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT

Sponsor:

Catalyst Pharmaceuticals, Inc.

Code:

NCT06564974

Conditions

Duchenne Muscular Dystrophy

Eligibility Criteria

Sex: Male

Age: 2+

Healthy Volunteers: Not accepted

Interventions

Vamorolone

Study Details

Brief summary:

The goal of this study is to collect additional information on the safety of long-term treatment with AGAMREE® and to explore long-term clinical impact of AGAMREE® on quality of life, as assessed by standardized patient-reported outcome measures (QoL questionnaires) in male patients aged 2 years and older with Duchenne muscular dystrophy (DMD).

Conditions

Duchenne Muscular Dystrophy

Study ID

NCT06564974

Start date

Sep 25, 2024

Status verified date

Sep, 2025

Completion date

Feb, 2032

Anticipated

Primary completion date

Feb, 2032

Anticipated

Eligibility Criteria

Sex: Male

Age: 2+

Healthy Volunteers: Not accepted

Inclusion Criteria:

1. Patient or parent/legal guardian is willing and able to provide written informed consent once the nature of the registry has been explained and prior to the start of any registry-related procedures.
2. Patient and/or parent/guardian are willing and able to complete QoL questionnaires.
3. Male patients at least 2 years old.
4. Confirmed diagnosis of DMD (via genetic testing or muscle biopsy with absent dystrophin staining to anti- dystrophin antibodies 3, 1, or 2, or dystrophin immunohistochemistry or western blot).
5. Patient has a current, active prescription for, or is on, AGAMREE®.

Exclusion Criteria:

1\. Any contraindication to AGAMREE® or medical condition, which, in the opinion of the Investigator, would affect registry participation, performance, or interpretation of registry assessments.

Study Design

Enrollment

250 participants

Anticipated

Interventions and Outcome Measures

Interventions

Vamorolone

Study treatment is AGAMREE®, which is commercially available as an oral suspension.

Primary outcome measure

Change in Height z-score [ Time Frame: At Enrollment Visit (baseline), and at each Yearly Follow-up Visit (for up to 5 years). ]
Change in BMI z-score [ Time Frame: At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years) ]
Change in North Star Ambulatory Assessment from baseline [ Time Frame: At Enrollment visit, and each Yearly Follow-up Visit (for up to 5 years). ]
Change in Performance of Upper Limb from baseline [ Time Frame: At Enrollment Visit, at each Yearly Follow-up Visit (for up to 5 years). ]
Tanner stage documentation [ Time Frame: At Enrollment Visit (baseline), at each Yearly Follow-up Visit (for up to 5 years). ]
Percentage of patients with cataract and/or glaucoma [ Time Frame: At Enrollment (baseline), and during each Yearly Follow-up Visit (for up to 5 years). ]
Findings from lateral spine x-ray [ Time Frame: At Enrollment Visit (baseline), and during each Yearly Follow-up Visit (for up to 5 years). ]
Findings from anterior-posterior/posterior-anterior X-ray [ Time Frame: At Enrollment Visit (baseline), and at each Yearly Follow-Up Visit (for up to 5 years). ]
Findings from hand-wrist X-ray [ Time Frame: At Enrollment Visit (baseline), and each Yearly Follow-Up Visit (for up to 5 years). ]
Summary statistics of bone mineral density measures assessed by dual x-ray absorptiometry [ Time Frame: At Enrollment Visit (baseline) and each Yearly Follow-Up Visit (for up to 5 years). ]
Body Composition Mass Measures [ Time Frame: At Enrollment Visit, each Yearly Follow-Up and End of Study/Early Termination (for approximately 5 years). ]
Summary statistics of body fat percentage and regional fat distribution ratios assessed by DXA [ Time Frame: At Enrollment visit, each Yearly Follow-up visit, and End of study/Early termination (for approximately 5 years). ]
Findings from standard of care echocardiography [ Time Frame: At Enrollment Visit, and at each Yearly Follow-up Visit (for up to 5 years). ]
Frequency and percentage of participants experiencing any adverse events and serious adverse events [ Time Frame: From informed consent, Enrollment visit, each Yearly follow-up visit, and End of Study/Early Termination (for up to 5 years). ]
Findings from cardiac magnetic resonance imaging (subset of patients taking part in the cardiac sub-study only) [ Time Frame: Enrollment Visit, and at each Yearly Follow-Up Visit (for up to 5 years). ]
Presence of cardiomyopathy assessed by echocardiography using transmural strain profile (only in a subset of patients taking part in the cardiac sub-study) [ Time Frame: At Enrollment Visit (baseline), and each Yearly Follow-up Visit (for up to 5 years) or Early Termination. ]

Central Contacts and Locations

Central contacts

Mark Wingertzahn, PhD

(305) 420-3200 mark.wingertzahn@catalystpharma.com

Locations

Phoenix Children's Hospital

Recruiting

Phoenix, Arizona, United States, 85016

Contacts

Saunder Bernes, MD

602 933 0956 sbernes@phoenixchildrens.com

Arkansas Childrens Hospital

Recruiting

Little Rock, Arkansas, United States, 72202

Contacts

Aravindhan Veerapandiyan, MD

(501) 364-1850 aveerapandiyan@uams.edu

Nicklaus Children's Hospital

Recruiting

Miami, Florida, United States, 33155

Contacts

Migvis Monduy, MD

305 663 8330 Migvis.Monduy@Nicklaushealth.org

Nemours Children's Hospital

Recruiting

Orlando, Florida, United States, 32827

Contacts

Omer Abdul Hamid, MD

407 650 7715 Omer.Abdulhamid@nemours.org

Lurie Children's Hospital of Chicago

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Nancy Kuntz, MD

312 227 4000 NKuntz@luriechildrens.org

Indiana University Health - Riley Hospital for Children

Recruiting

Indianapolis, Indiana, United States, 46202

Contacts

Marcia Felker, MD

317 278 7364 mamccann@iu.edu

University of Kansas Medical Center

Recruiting

Kansas City, Kansas, United States, 66205

Contacts

Jeffrey Statland, MD

913 588 6970 JStatland@kumc.edu

University of Massachusetts Memorial Medical Center

Recruiting

North Worcester, Massachusetts, United States, 01655

Contacts

Stephen M. Chrzanowski, MD, PhD

(774) 441-7615 Stephen.Chrzanowski@umassmed.edu

Helen DeVos Children's Hospital

Recruiting

Grand Rapids, Michigan, United States, 49503

Contacts

Jena Krueger, MD

616-447-5836 Jena.Krueger@helendevoschildrens.org

Atrium Health Neurosciences Institute

Recruiting

Charlotte, North Carolina, United States, 28207

Contacts

Urvi Desai, MD

704 468 0101 Urvi.Desai@atriumhealth.org

Duke University Medical Center and Childrens Health Center

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Mai ElMallah, MD

(919) 681-3364 mai.elmallah@duke.edu

Cincinnati Children's Hospital Medical Center

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Meghan McAnally, MD

513 636 9285 meghan.mcanally@cchmc.org

Penn State Milton S. Hershey Medical Center- Penn State Hershey Neuroscience Institute

Recruiting

Hershey, Pennsylvania, United States, 17033

Contacts

Dustin Paul, MD

717-531-3828 dpaul2@pennstatehealth.psu.edu

Children's Hospital of Philadelphia (CHOP)

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Sabrina Yum, MD

215 590 4719 YUMS@chop.edu

University of Texas Southwestern Medical Center

Recruiting

Dallas, Texas, United States, 75235

Contacts

Kaitlin Batley, MD

214 456 9561 Kaitlin.Batley@UTSouthwestern.edu

Neurology Rare Disease Center - Neurology & Neuromuscular Care Center

Recruiting

Denton, Texas, United States, 76208

Contacts

Diana Castro, MD

972 982 7411 Research@neuromdcenter.com

The University of Texas Health Science Center at San Antonio

Recruiting

San Antonio, Texas, United States, 78229

Contacts

Matthew Wicklund, MD

210 450 0500 wicklund@uthscsa.edu

University of Virginia Health System (UVAHS) - Pediatric Neuromuscular Center

Recruiting

Charlottesville, Virginia, United States, 22903

Contacts

Rebecca Scharf, MD

434 924 1647 RS3YK@uvahealth.org

Seattle Children's Hospital

Recruiting

Seattle, Washington, United States, 98105

Contacts

Seth Perlman, MD

206-987-0804 Seth.Perlman@seattlechildrens.org

More Information

Sponsor

Catalyst Pharmaceuticals, Inc.

Last update posted

Jul 20, 2026

Last verified

Sep, 2025

Keywords

Duchenne Muscular Dystrophy
Neuromuscular
AGAMREE®
Vamorolone
DMD
Muscular Dystrophy

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Catalyst Pharmaceuticals, Inc. on 2026-07-20.